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Intraputamenal Cere...
Intraputamenal Cerebral Dopamine Neurotrophic Factor in Parkinson's Disease: A Randomized, Double-Blind, Multicenter Phase 1 Trial
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- Huttunen, Henri J. (författare)
- Herantis Pharma Plc, Espoo, Finland
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- Booms, Sigrid (författare)
- Herantis Pharma Plc, Espoo, Finland
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- Sjögren, Magnus (författare)
- Umeå universitet,Psykiatri,Herantis Pharma Plc, Espoo, Finland
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- Kerstens, Vera (författare)
- Department of Clinical Neuroscience, Centre for Psychiatry Research, Karolinska Institutet and Stockholm Health Care Services, Stockholm, Sweden
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- Johansson, Jarkko (författare)
- Umeå universitet,Umeå centrum för funktionell hjärnavbildning (UFBI),Institutionen för strålningsvetenskaper
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- Holmnäs, Rebecka (författare)
- Herantis Pharma Plc, Espoo, Finland
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- Koskinen, Jani (författare)
- Herantis Pharma Plc, Espoo, Finland
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- Kulesskaya, Natalia (författare)
- Herantis Pharma Plc, Espoo, Finland
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- Fazio, Patrik (författare)
- Department of Clinical Neuroscience, Centre for Psychiatry Research, Karolinska Institutet and Stockholm Health Care Services, Stockholm, Sweden; Department of Neurology, Karolinska University Hospital, Stockholm, Sweden
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- Woolley, Max (författare)
- Renishaw Neuro Solutions Ltd, Gloucestershire, United Kingdom
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- Brady, Alan (författare)
- Renishaw Neuro Solutions Ltd, Gloucestershire, United Kingdom
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- Williams, Julia (författare)
- Renishaw Neuro Solutions Ltd, Gloucestershire, United Kingdom
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- Johnson, David (författare)
- Renishaw Neuro Solutions Ltd, Gloucestershire, United Kingdom
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- Dailami, Narges (författare)
- Renishaw Neuro Solutions Ltd, Gloucestershire, United Kingdom; Department of Computer Science and Creative Technology, University of the West of England, Bristol, United Kingdom
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- Gray, William (författare)
- Renishaw Neuro Solutions Ltd, Gloucestershire, United Kingdom; Functional Neurosurgery, Neuroscience and Mental Health Innovation Institute, Cardiff University, Cardiff, United Kingdom
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- Levo, Reeta (författare)
- Department of Neurology, Helsinki University Hospital, Helsinki, Finland; Clinicum, University of Helsinki, Helsinki, Finland
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- Saarma, Mart (författare)
- Institute of Biotechnology, HiLIFE, University of Helsinki, Helsinki, Finland
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- Halldin, Christer (författare)
- Karolinska Institutet
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- Marjamaa, Johan (författare)
- Clinicum, University of Helsinki, Helsinki, Finland; Department of Neurosurgery, Helsinki University Hospital, Helsinki, Finland
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- Resendiz-Nieves, Julio (författare)
- Clinicum, University of Helsinki, Helsinki, Finland; Department of Neurosurgery, Helsinki University Hospital, Helsinki, Finland
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- Grubor, Irena (författare)
- Department of Neurosurgery, Skåne University Hospital, Lund, Sweden
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- Lind, Göran (författare)
- Department of Neurosurgery, Karolinska University Hospital, Stockholm, Sweden; Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden
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- Eerola-Rautio, Johanna (författare)
- Department of Neurology, Helsinki University Hospital, Helsinki, Finland; Clinicum, University of Helsinki, Helsinki, Finland
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- Mertsalmi, Tuomas (författare)
- Department of Neurology, Helsinki University Hospital, Helsinki, Finland; Clinicum, University of Helsinki, Helsinki, Finland
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- Andréasson, Mattias (författare)
- Karolinska Institutet
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- Paul, Gesine (författare)
- Department of Neurology, Skåne University Hospital, Lund, Sweden
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- Rinne, Juha (författare)
- Turku PET Centre, University of Turku and Turku University Hospital, Turku, Finland
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- Kivisaari, Riku (författare)
- Clinicum, University of Helsinki, Helsinki, Finland; Department of Neurosurgery, Helsinki University Hospital, Helsinki, Finland
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- Bjartmarz, Hjalmar (författare)
- Department of Neurosurgery, Skåne University Hospital, Lund, Sweden
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- Almqvist, Per (författare)
- Department of Neurosurgery, Karolinska University Hospital, Stockholm, Sweden; Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden
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- Varrone, Andrea (författare)
- Karolinska Institutet
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- Scheperjans, Filip (författare)
- Department of Neurology, Helsinki University Hospital, Helsinki, Finland; Clinicum, University of Helsinki, Helsinki, Finland
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- Widner, Håkan (författare)
- Department of Neurology, Skåne University Hospital, Lund, Sweden
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- Svenningsson, Per (författare)
- Karolinska Institutet
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(creator_code:org_t)
- John Wiley & Sons, 2023
- 2023
- Engelska.
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Ingår i: Movement Disorders. - : John Wiley & Sons. - 0885-3185 .- 1531-8257. ; 38:7, s. 1209-1222
- Relaterad länk:
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https://doi.org/10.1...
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https://umu.diva-por... (primary) (Raw object)
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https://urn.kb.se/re...
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https://doi.org/10.1...
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http://kipublication...
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Abstract
Ämnesord
Stäng
- Background: Cerebral dopamine neurotrophic factor (CDNF) is an unconventional neurotrophic factor that protects dopamine neurons and improves motor function in animal models of Parkinson's disease (PD). Objective: The primary objectives of this study were to assess the safety and tolerability of both CDNF and the drug delivery system (DDS) in patients with PD of moderate severity. Methods: We assessed the safety and tolerability of monthly intraputamenal CDNF infusions in patients with PD using an investigational DDS, a bone-anchored transcutaneous port connected to four catheters. This phase 1 trial was divided into a placebo-controlled, double-blind, 6-month main study followed by an active-treatment 6-month extension. Eligible patients, aged 35 to 75 years, had moderate idiopathic PD for 5 to 15 years and Hoehn and Yahr score ≤ 3 (off state). Seventeen patients were randomized to placebo (n = 6), 0.4 mg CDNF (n = 6), or 1.2 mg CDNF (n = 5). The primary endpoints were safety and tolerability of CDNF and DDS and catheter implantation accuracy. Secondary endpoints were measures of PD symptoms, including Unified Parkinson's Disease Rating Scale, and DDS patency and port stability. Exploratory endpoints included motor symptom assessment (PKG, Global Kinetics Pty Ltd, Melbourne, Australia) and positron emission tomography using dopamine transporter radioligand [18F]FE-PE2I. Results: Drug-related adverse events were mild to moderate with no difference between placebo and treatment groups. No severe adverse events were associated with the drug, and device delivery accuracy met specification. The severe adverse events recorded were associated with the infusion procedure and did not reoccur after procedural modification. There were no significant changes between placebo and CDNF treatment groups in secondary endpoints between baseline and the end of the main and extension studies. Conclusions: Intraputamenally administered CDNF was safe and well tolerated, and possible signs of biological response to the drug were observed in individual patients. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Neurologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Neurology (hsv//eng)
Nyckelord
- clinical trial
- convection-enhanced delivery
- movement disorder
- neurotrophic factor
- synucleinopathy
- transcutaneous port
Publikations- och innehållstyp
- ref (ämneskategori)
- art (ämneskategori)
Hitta via bibliotek
Till lärosätets databas
- Av författaren/redakt...
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Huttunen, Henri ...
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Booms, Sigrid
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Sjögren, Magnus
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Kerstens, Vera
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Johansson, Jarkk ...
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Holmnäs, Rebecka
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visa fler...
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Koskinen, Jani
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Kulesskaya, Nata ...
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Fazio, Patrik
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Woolley, Max
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Brady, Alan
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Williams, Julia
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Johnson, David
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Dailami, Narges
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Gray, William
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Levo, Reeta
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Saarma, Mart
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Halldin, Christe ...
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Marjamaa, Johan
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Resendiz-Nieves, ...
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Grubor, Irena
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Lind, Göran
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Eerola-Rautio, J ...
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Mertsalmi, Tuoma ...
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Andréasson, Matt ...
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Paul, Gesine
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Rinne, Juha
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Kivisaari, Riku
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Bjartmarz, Hjalm ...
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Almqvist, Per
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Varrone, Andrea
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Scheperjans, Fil ...
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Widner, Håkan
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Svenningsson, Pe ...
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- Om ämnet
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Neurologi
- Artiklar i publikationen
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Movement Disorde ...
- Av lärosätet
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Umeå universitet
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Karolinska Institutet